PENN NEURODEGENERATION GENOMICS CENTER: SEMINAR SERIESApril 5, 2017 - 2:00pm - John Morgan Reunion AuditoriumJustin Ichida, PhD — "Identification of therapeutic targets for ALS using chemical and genetic screens on patient-derived neural cultures" Abstract:Neurodegenerative disease is one of the only causes of human morbidity that continues to increase in lethality. Decades of drug development efforts suggest that new therapeutic targets and a better understanding of disease processes will be necessary for curative treatments. To this end, we have employed iPSC technology to develop a phenotypic screen for ALS usingC9ORF72and sporadic patient neural cultures. We have identified new targets that when perturbed by small molecules can rescue the neurodegenerative processes inC9ORF72ALS patient motor neurons. In addition, we have identified new genetic mutations that may contribute to sporadic ALS, some of which converge mechanistically with targets identified by our small molecule screens onC9ORF72patient motor neurons. Combined with mechanistic studies in iPSC-derived cultures, post-mortem tissue, and mice, these studies elucidate ALS disease processes and point to new therapeutic targets that may modify disease progression in humans.